A new medication to reduce the frequency of painful crises in sickle cell disease is expected to be available to patients within weeks.
Two months ahead of its scheduled action date, the FDA approved crizanlizumab (Adakveo, Novartis) to reduce the frequency of vaso-occlusive crises (VOCs) in patients 16 years of age and older. Crizanlizumab is the first FDA-approved sickle cell medicine that binds to P-selectin—a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion.
FDA approval is based on results of the 52-week, randomized, placebo-controlled SUSTAIN trial, which showed that crizanlizumab significantly lowered the median annual rate of VOCs to 1.63 versus 2.98 with placebo—equivalent to a 45% reduction. Reductions in the frequency of VOCs were seen among patients regardless of sickle cell disease genotype or hydroxyurea use.
“The approval of crizanlizumab is an important advancement for people living with this very difficult condition,” said Kenneth Ataga, MD, Director of the Center for Sickle Cell Disease at the University of Tennessee Health Science Center at Memphis and Principal Investigator of the SUSTAIN trial.
In that trial, the median annual rate of days hospitalized fell 42% with crizanlizumab compared with placebo (4 days versus 6.87 days). Thirty-six percent of patients treated with crizanlizumab did not experience a VOC, compared to 17% of placebo-treated patients. The median time to first VOC was 4.1 months for crizanlizumab versus 1.4 months for placebo. The most common adverse reactions were nausea, arthralgia, back pain, and pyrexia.
The drug is administered on the basis of weight, 5 mg/kg, over a period of 30 minutes by intravenous infusion on week 0, week 2, and every four weeks thereafter.
Considered the clinical hallmark of the disease, sickle cell pain crises are triggered, in part, by multicellular interactions that form clusters of cells, which can block or reduce the blood flow to organs. Sickle cell pain crises can be frequent and sudden; they are associated with an increased risk of life-threatening complications. They are also the main reason why individuals living with sickle cell disease go to the emergency room and are admitted to the hospital.
Source: Novartis, November 15, 2019